Smallpox Treatment Receives Orphan Drug Designation From FDA

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for brincidofovir for the treatment of smallpox, a serious, and often deadly infectious disease.

Since 2011, Chimerix has been working closely with the Biomedical Advanced Research and Development Authority to develop the experimental antiviral drug as a medical countermeasure to treat the disease and any potential outbreaks that may spring up due to a bioterror event or even accidental release.

Brincidofovir (CMX001) has shown to demonstrate improved survival rates following orthopoxvirus infections in several animal models.

“We are very pleased to have received Orphan Drug Designation from the FDA for brincidofovir as a treatment for smallpox,” Chimerix’s president and chief executive officer M Michelle Berrey, MD, MPH, said in a recent statement. “Though declared eradicated in the late 1970s, smallpox, whether natural of synthetic, continues to be a potential global threat in today’s interconnected world.”

Although the last natural outbreak of virus occurred back in 1949, it seems that as the frozen Siberian tundra continues to melt, the chances of smallpox making a comeback may not be so far-fetched.

For example, experts warn that as the tundra melts, corpses of those who died during an epidemic that occurred over 120 years ago may resurface, and if that happens, the disease might as well. In addition, in Canada, a team of researchers successfully synthesized the horsepox virus, which is a close relative of smallpox. Experts in the field highlighted this experiment as a possible opening of Pandora’s box when it comes to risky research that could potentially result in disease re-emergence.

Previous to this ODD, the FDA accepted SIGA Technologies, Inc’s New Drug Application for an oral formulation of a smallpox medication called tecovirimat (TPOXX), a novel small molecule antiviral therapy. They also granted priority review to the application, with the target final action date identified to be August 8, 2018.

The ODD program provides orphan drug status to drugs and biologics defined as those that are intended for safe and effective treatment, diagnosis, or prevention of rare diseases and disorders that affect less than 200,000 individuals in the United States.

“We are committed to completing the development program for brincidofovir as a much-needed treatment option for smallpox, in close collaboration with BARDA and FDA,” Dr Berrey said.